CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE:
4502) today announced that the European Commission has granted
conditional marketing authorization for NINLAROTM (ixazomib)
capsules, indicated in combination with lenalidomide and dexamethasone
for adult patients with multiple myeloma who have received at least one
prior therapy. The decision to approve NINLARO as the first and only
oral proteasome inhibitor to treat multiple myeloma follows a positive
opinion by the European Medicines Agency (EMA) Committee for Medicinal
Products (CHMP) for Human Use in September 2016.

“In
the TOURMALINE-MM1 study, we saw a clinically meaningful six-month
improvement in progression-free survival with NINLARO, evidence that has
supported its approval in Europe. As a hematologist, I welcome the
availability of this treatment to address a devastating disease like
multiple myeloma.”

“For myeloma patients living in Europe, the approval of NINLARO means we
have a new and effective treatment option available when we relapse,”
said Bob Munro, a patient with multiple myeloma from the United Kingdom.
“I applaud the European Commission for recognising the additional
benefit that NINLARO will bring to patients, who not only want treatment
options that are effective and tolerable, but also appreciate the
convenient option of taking an oral treatment. I strongly hope this will
be made available by national health systems across Europe as soon as
possible.”

The European Commission followed the CHMP’s recommendation to approve
NINLARO based on data from the pivotal Phase 3 TOURMALINE-MM1 trial,
which demonstrated that NINLARO plus lenalidomide and dexamethasone
increased the length of progression-free survival by about six months,
or 40 percent, in patients with relapsed and refractory multiple myeloma
when compared with placebo, lenalidomide and dexamethasone. The study
also showed that the progression-free survival benefit observed in the
NINLARO regimen extended across pre-specified subgroups of patients.
Follow-up analyses for overall survival are planned for 2017.

“With the approval of NINLARO by the European Commission, physicians
across the region will have the option to prescribe an all-oral triplet
regimen to treat patients with multiple myeloma who have received at
least one prior therapy,” said Philippe Moreau, MD, Head of the
Hematology Department at the University Hospital of Nantes, France. “In
the TOURMALINE-MM1 study, we saw a clinically meaningful six-month
improvement in progression-free survival with NINLARO, evidence that has
supported its approval in Europe. As a hematologist, I welcome the
availability of this treatment to address a devastating disease like
multiple myeloma.”

“When developing NINLARO, Takeda Oncology’s scientists sought to
formulate an efficacious and unique oral proteasome inhibitor with a
manageable safety profile. NINLARO delivers the proven efficacy of a
proteasome inhibitor in a convenient once-weekly pill that can be taken
at home,” said Christophe Bianchi, M.D., President, Takeda Oncology.
“NINLARO has the potential to help European patients with relapsed
multiple myeloma by removing some of the barriers that can stand in the
way of optimal treatment. With NINLARO, our hope is that many patients
will be able to continue therapy until disease progression. Following
the European Commission’s approval, we will continue to study NINLARO in
a variety of settings in the hopes that we can bring this medicine to as
many of the patients who may benefit from it as possible.”

As a result of the European Commission decision, NINLARO is now approved
for use across the European Economic Area, which includes the European
Union’s 28 member states as well as Norway, Liechtenstein and Iceland.
In addition, NINLARO is licensed for use in the U.S., Canada, Israel,
Australia and Venezuela, and Takeda has submitted marketing
authorization applications for NINLARO to a number of additional
regulatory authorities around the world.

About NINLAROTM(ixazomib) capsules

NINLAROTM(ixazomib) is an oral proteasome inhibitor
which is also being studied across the continuum of multiple myeloma
treatment settings as well as systemic light-chain (AL) amyloidosis. It
was the first oral proteasome inhibitor to enter Phase 3 clinical trials
and to receive approval. NINLARO was approved by the U.S. Food and Drug
Administration (FDA) in November 2015 following a priority review. In
the U.S., NINLARO is indicated in combination with lenalidomide and
dexamethasone for the treatment of patients with multiple myeloma who
have received at least one prior therapy.

Ixazomib was granted orphan drug designation in multiple myeloma in both
the U.S. and Europe in 2011 and for AL amyloidosis in both the U.S. and
Europe in 2012. Ixazomib received Breakthrough Therapy status by the
U.S. FDA for relapsed or refractory systemic light-chain (AL)
amyloidosis in 2014.

The comprehensive ixazomib clinical development program, TOURMALINE,
further reinforces Takeda’s ongoing commitment to developing innovative
therapies for people living with multiple myeloma worldwide and the
healthcare professionals who treat them. TOURMALINE includes a total of
five ongoing pivotal trials – four, which together are investigating
every major multiple myeloma patient population, and one in light-chain
amyloidosis:

In addition to the TOURMALINE program, ixazomib is being evaluated in
multiple therapeutic combinations for various patient populations in
investigator initiated studies globally.

NINLAROTM (ixazomib): Global Important Safety
Information

SPECIAL WARNINGS AND PRECAUTIONS

Thrombocytopenia has been reported with NINLARO (28% vs. 14% in
the NINLARO and placebo regimens, respectively) with platelet nadirs
typically occurring between Days 14-21 of each 28-day cycle and recovery
to baseline by the start of the next cycle. It did not result in an
increase in hemorrhagic events or platelet transfusions. Monitor
platelet counts at least monthly during treatment with NINLARO and
consider more frequent monitoring during the first three cycles. Manage
with dose modifications and platelet transfusions as per standard
medical guidelines.

Peripheral neuropathy was reported with NINLARO (28% vs. 21% in
the NINLARO and placebo regimens, respectively). The most commonly
reported reaction was peripheral sensory neuropathy (19% and 14% in the
NINLARO and placebo regimens, respectively). Peripheral motor neuropathy
was not commonly reported in either regimen (< 1%). Monitor patients for
symptoms of peripheral neuropathy and adjust dosing as needed.

Peripheral edema was reported with NINLARO (25% vs. 18% in the
NINLARO and placebo regimens, respectively). Evaluate patients for
underlying causes and provide supportive care, as necessary. Adjust the
dose of dexamethasone per its prescribing information or the dose of
NINLARO for severe symptoms.

Cutaneous reactions occurred in 19% of patients in the NINLARO
regimen compared to 11% of patients in the placebo regimen. The most
common type of rash reported in both regimens was maculo-papular and
macular rash. Manage rash with supportive care, dose modification or
discontinuation.

Pregnancy- NINLARO can cause fetal harm. Advise male and females
patients of reproductive potential to use contraceptive measures during
treatment and for an additional 90 days after the final dose of NINLARO.
Women of childbearing potential should avoid becoming pregnant while
taking NINLARO due to potential hazard to the fetus. Women using
hormonal contraceptives should use an additional barrier method of
contraception.

Lactation- It is not known whether NINLARO or its metabolites are
excreted in human milk. There could be potential adverse events in
nursing infants and therefore breastfeeding should be discontinued.

Multiple myeloma is a cancer of the plasma cells, which are found in the
bone marrow. In multiple myeloma, a group of monoclonal plasma cells, or
myeloma cells, becomes cancerous and multiplies. These malignant plasma
cells have the potential to affect many bones in the body, possibly
resulting in compression fractures, lytic bone lesions and related pain.
Multiple myeloma can cause a number of serious health problems affecting
the bones, immune system, kidneys and red blood cell count, with some of
the more common symptoms including bone pain and fatigue, a symptom of
anemia. Multiple myeloma is a rare form of cancer, with approximately
39,000 new cases in the EU and 114,000 new cases globally per year.

About Takeda Pharmaceutical Company

Takeda Pharmaceutical Company Limited is a global, research and
development-driven pharmaceutical company committed to bringing better
health and a brighter future to patients by translating science into
life-changing medicines. Takeda focuses its R&D efforts on oncology,
gastroenterology and central nervous system therapeutic areas plus
vaccines. Takeda conducts R&D both internally and with partners to stay
at the leading edge of innovation. New innovative products, especially
in oncology and gastroenterology, as well as our presence in Emerging
Markets, fuel the growth of Takeda. More than 30,000 Takeda employees
are committed to improving quality of life for patients, working with
our partners in health care in more than 70 countries. For more
information, visit http://www.takeda.com/news.

Additional information about Takeda is available through its corporate
website, www.takeda.com,
and additional information about Takeda Oncology, the brand for the
global oncology business unit of Takeda Pharmaceutical Company Limited,
is available through its website, www.takedaoncology.com.